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ECSP18010463A - COMPOSITIONS OF OLIGONUCLEOTIDES AND METHODS OF THE SAME - Google Patents

COMPOSITIONS OF OLIGONUCLEOTIDES AND METHODS OF THE SAME

Info

Publication number
ECSP18010463A
ECSP18010463A ECIEPI201810463A ECPI201810463A ECSP18010463A EC SP18010463 A ECSP18010463 A EC SP18010463A EC IEPI201810463 A ECIEPI201810463 A EC IEPI201810463A EC PI201810463 A ECPI201810463 A EC PI201810463A EC SP18010463 A ECSP18010463 A EC SP18010463A
Authority
EC
Ecuador
Prior art keywords
chirally controlled
oligonucleotide composition
controlled oligonucleotide
modifications
methods
Prior art date
Application number
ECIEPI201810463A
Other languages
Spanish (es)
Inventor
Anna Sokolovska
Meena
Gregory L Verdine
Christopher J Francis
Susovan Mohapatra
Nenad Svrzikapa
Chandra Vargeese
Original Assignee
Wave Life Sciences Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Wave Life Sciences Ltd filed Critical Wave Life Sciences Ltd
Publication of ECSP18010463A publication Critical patent/ECSP18010463A/en

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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

Entre otras cosas, la presente divulgación se refiere a oligonucleótidos quiralmente controlados de diseños seleccionados, composiciones oligonucleotídicas quiralmente controladas y métodos de preparación y uso de ellos. En algunas formas de realización, una composición oligonucleotídica quiralmente controlada suministrada proporciona diferentes patrones de escisión de un polímero de ácido nucleico que una composición oligonucleotídica de referencia. En algunas formas de realización, una composición oligonucleotídica quiralmente controlada suministrada proporciona escisión de sitio único dentro de una secuencia complementaria de un polímero de ácido nucleico. En algunas formas de realización, una composición oligonucleotídica quiralmente controlada tiene cualquier secuencia de bases, y/o patrón o modificaciones de base, modificaciones de azúcar, modificaciones estructurales y/o estereoquímica o combinación de estos elementos descritos en la presente.Among other things, the present disclosure relates to chirally controlled oligonucleotides of selected designs, chirally controlled oligonucleotide compositions, and methods of preparing and using them. In some embodiments, a supplied chirally controlled oligonucleotide composition provides different cleavage patterns from a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a supplied chirally controlled oligonucleotide composition provides for single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and / or base pattern or modifications, sugar modifications, structural and / or stereochemical modifications, or combinations of these elements described herein.

ECIEPI201810463A 2015-07-22 2018-02-09 COMPOSITIONS OF OLIGONUCLEOTIDES AND METHODS OF THE SAME ECSP18010463A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562195779P 2015-07-22 2015-07-22
US201562236847P 2015-10-02 2015-10-02
US201662331960P 2016-05-04 2016-05-04

Publications (1)

Publication Number Publication Date
ECSP18010463A true ECSP18010463A (en) 2018-04-30

Family

ID=57835219

Family Applications (1)

Application Number Title Priority Date Filing Date
ECIEPI201810463A ECSP18010463A (en) 2015-07-22 2018-02-09 COMPOSITIONS OF OLIGONUCLEOTIDES AND METHODS OF THE SAME

Country Status (23)

Country Link
US (6) US20180216107A1 (en)
EP (3) EP4344744A3 (en)
JP (6) JP2018525357A (en)
KR (1) KR20180028516A (en)
CN (3) CN117965536A (en)
AU (3) AU2016297155B2 (en)
BR (1) BR112017028636A2 (en)
CA (1) CA2989682A1 (en)
CL (1) CL2018000145A1 (en)
CO (1) CO2018001484A2 (en)
CR (1) CR20180107A (en)
EC (1) ECSP18010463A (en)
HK (2) HK1255370A1 (en)
IL (2) IL320434A (en)
MA (1) MA43072A (en)
MX (2) MX2018000796A (en)
NI (1) NI201800009A (en)
PE (1) PE20181174A1 (en)
PH (1) PH12018500145A1 (en)
SG (1) SG10201912900XA (en)
SV (1) SV2018005619A (en)
TW (1) TW201707711A (en)
WO (2) WO2017015575A1 (en)

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